那他珠单抗成功治疗两名Susac综合征女性患者。
Successful Natalizumab Treatment of Two Female Individuals With Susac Syndrome.
作者信息
Konitsioti Agni M, Grajewski Rafael, Schlamann Mark, Schroeter Michael, Fink Gereon R, Warnke Clemens
机构信息
Department of Neurology, University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany.
Department of Ophthalmology, Faculty of Medicine and University Hospital of Cologne, Cologne, Germany.
出版信息
Eur J Neurol. 2025 Mar;32(3):e70103. doi: 10.1111/ene.70103.
BACKGROUND
Susac syndrome is a rare autoimmune endotheliopathy that affects the central nervous system, retina, and inner ear, characterized by encephalopathy, branch retinal artery occlusions, and sensorineural hearing loss. Due to the heterogeneity of its presentation, early diagnosis, and treatment remain challenging.
OBJECTIVE/METHODS: To evaluate the clinical outcomes and radiological responses in two patients with Susac syndrome treated with natalizumab in an off-label therapeutic approach, clinical assessments and serial magnetic resonance imaging (MRI) were performed over a follow-up period of up to 22 months to monitor disease progression and treatment response.
RESULTS
Both patients demonstrated clinical stabilization with reduced MRI and retinal angiography disease activity. Treatment was well tolerated, and no significant adverse events were reported during observation.
DISCUSSION
Natalizumab may constitute a potential off-label therapeutic for Susac syndrome. Further studies are warranted to assess its efficacy and safety in this rare condition.
背景
Susac综合征是一种罕见的自身免疫性内皮病，累及中枢神经系统、视网膜和内耳，其特征为脑病、视网膜分支动脉阻塞和感音神经性听力损失。由于其临床表现具有异质性，早期诊断和治疗仍然具有挑战性。
目的/方法：为了评估两名采用那他珠单抗进行超说明书治疗的Susac综合征患者的临床结局和影像学反应，在长达22个月的随访期内进行了临床评估和系列磁共振成像（MRI）检查，以监测疾病进展和治疗反应。
结果
两名患者均表现出临床病情稳定，MRI及视网膜血管造影显示疾病活动度降低。治疗耐受性良好，观察期间未报告重大不良事件。
讨论
那他珠单抗可能构成Susac综合征的一种潜在超说明书治疗方法。有必要进一步开展研究以评估其在这种罕见疾病中的疗效和安全性。
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